| S6003 |
Ataluren (PTC124)
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Ataluren (PTC124) selectively induces ribosomal read-through of premature but not normal termination codons, with EC50 of 0.1 μM in HEK293 cells, and may provide treatment for genetic disorders caused by nonsense mutations (e.g. CF caused by CFTR nonsense mutation). This compound is currently in Phase 3.
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Int J Mol Med, 2025, 55(5)69
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Stem Cell Reports, 2022, 17(10):2187-2202
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Int J Mol Sci, 2022, 23(7)3541
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| S7139 |
CFTRinh-172
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CFTRinh-172 (CFTR inhibitor 172) is a voltage-independent, selective CFTR inhibitor with Ki of 300 nM, showing no effects on MDR1, ATP-sensitive K+ channels, or a series of other transporters.
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Mol Ther Nucleic Acids, 2025, 36(4):102756
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J Cyst Fibros, 2025, S1569-1993(25)01612-1
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Nat Cancer, 2024, 10.1038/s43018-024-00731-2
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| S8094 |
GlyH-101
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GlyH-101 is a selective and reversible CFTR inhibitor with Ki of 4.3 μM.
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mBio, 2023, 14(5):e0151623
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Cell Mol Immunol, 2020, 10.1038/s41423-020-0499-3
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| S7329 |
IOWH032
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IOWH032 is a synthetic CFTR inhibitor with IC50 of 1.01 μM in CHO-CFTR cell based assays. Phase 2.
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Sci Rep, 2023, 13(1):3934
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| S0198 |
PPQ-102
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PPQ-102 (CFTR Inhibitor IV) is a potent inhibitor of CFTR. This compound can completely inhibit CFTR chloride current with IC50 of ~90 nM.
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Cells, 2023, 12(5)776
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| S3272 |
Steviol (Hydroxydehydrostevic acid)
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Steviol (Hydroxydehydrostevic acid, Hydroxy Dehydrostevic Acid, NSC 226902), a major metabolite of the sweetening compound stevioside, inhibits CFTR activity, reduces AQP2 expression and promotes AQP2 degradation.
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